A study suggests that the availability of orphan drugs varies significantly between different countries in Europe.
Conducted by the University of Finland and published in the European Journal of Clinical Pharmacology, the study focused on the availability and distribution of ten orphan medicines used in outpatient care in 24 European countries.
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An orphan drug is a pharmaceutical product developed specifically to treat, prevent or diagnose a rare disease. Such drugs are often developed not for commercial reasons, but to meet a specific public health need.
In the US and the EU, it is easier to gain marketing approval for an orphan drug, and there may be other incentives, such as an extended period of exclusivity, intended to encourage the development of drugs for rare conditions that would otherwise lack a sufficient financial incentive.
Although the European Medicines Agency has policies in place to facilitate the development and authorisation of medicines for rare diseases, the study found that, on average, only five of the ten medicines included in the study were available on the markets, but there was variation between the countries. This suggests that although drugs for such conditions may exist, there may be barriers to accessing them in different European countries.
All ten medicines were available only in three countries: the Netherlands, Malta and Poland. Five to nine medicines were available in ten countries: Austria, Finland, Germany, Iceland, Italy, Norway, Slovakia, Spain, Sweden and the UK. Four countries—Latvia, Lithuania, Turkey and Belarus—did not have any of the medicines studied available.
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By GlobalDataThe study also explored whether the European countries studied had implemented policies to regulate decisions relating to the pricing and reimbursement of rare medicines specifically. Orphan drugs often come with a high price tag due to small patient populations and the high cost of development.
The study found that typically no policies applicable to rare medicines alone were in place; instead, decisions on rare medicines were made on the same grounds as decisions on other medicines. This calls into question whether orphan medicines should be considered as a separate group in the reimbursement regulations in order to secure patient access to these medicines.
The study was carried out in April 2016 via a survey sent to the members of a network of authorities dealing with pharmaceutical product pricing and reimbursement in Europe. The study was funded by Kela, the Social Insurance Institution of Finland, and the University of Eastern Finland.
